Metabolic Lab · DeCure for X

DeCure for Inherited lipid metabolism disorder

DeCure's Metabolic AI scientist maps inherited lipid metabolism disorder for drug repurposing — screening already-approved drugs against its 9-gene Open Targets disease module to publish open-access research. The community decides which DeCure goes to the lab first.

Disease module9 genesLead labMetabolic
All DeCures
MetabolicDOID:3146$DeCureMetabolic

The disease map

Disease moduleInherited lipid metabolism disorder maps to a 9-gene Open Targets module — the target space DeCure's AI scientist screens approved drugs against.
DeCure.ai methodSignature reversal (LINCS) plus network proximity (STRING) rank already-approved drugs likely to perturb this module — the same engine that produces DeCure.ai's repurposing hypotheses.
Repurposing thesisScreening approved medicines against this disease module, then publishing the evidence for the strongest candidate. Known pharmacology and human exposure data make the first question sharper — they do not establish safety or efficacy in a new indication.

Research record

01
ResearchComing soon
Candidate research + dossier — target rationale, drug-repurposing thesis and evidence pack.proof: Published dossier + on-chain hash
02
ValidationComing soon
In-vitro biological validation at a contract research org (CRO).proof: CRO contract + in-vitro report
03
Peer review & paperComing soon
Peer-reviewed paper published open-access (preprint + journal).proof: DOI + open-access link + on-chain hash

Drugs in the evidence

No approved-drug candidate for inherited lipid metabolism disorder is corroborated in the literature DeepSearch retrieved. Some conditions are managed with non-pharmacological care — a device, surgery or physical therapy — rather than a medicine; that may be the case here, or the literature we found may simply be too sparse yet to support a drug-repurposing angle.

Molecular view

3-hydroxy-3-methylglutaryl-CoA reductase (HMGCR) — HMGCR is one of the genes genetically linked to this disease in Open Targets — shown as context, not as a drug target we're pursuing: no approved-drug candidate for this disease is yet corroborated in the literature we found.

Loading structure…
helix sheet coadrag to rotate · scroll to zoom

RCSB Protein Data Bank · entry 1DQA · 2.0 Å · ligand COENZYME A (COA). Experimental structure, not a prediction.

Evidence

Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.

The FASEB Journal · 1995 · 17 citations

Physological significance of apolipoprotein mutants

AbstractThe major plasma lipids, cholesterol, and triglycerides are transported in the blood by different classes of lipoprotein, which can be differentiated from each other by their apoprotein and lipid constituents. Defects in the genes coding for the apolipoprotein components, besides those coding for lipolytic enzymes and cellular receptors, can cause an imbalance in the plasma lipid homeostasis. The physiological significance on lipid metabolism of genetic defects, both rare inborn errors or common genetic variation at these gene loci, are the topics of this review.

https://doi.org/10.1096/fasebj.9.9.7601341
Clinical Pediatric Endocrinology · 2019 · 7 citations · open access

The long-term management of congenital generalized lipodystrophy (Berardinelli-Seip syndrome): the clinical manifestations of Japanese siblings for approximately 20 years

AbstractCongenital generalized lipodystrophy (CGL) is a rare autosomal recessive disease that is characterized by loss of subcutaneous and visceral adipose tissues, and associated with dysregulation of glycolipid metabolism. In the present study, we reported the clinical manifestations and treatments of Japanese siblings with CGL caused by BSCL2 gene mutations with a clinical course of approximately 20 yr. Comprehensive management with metreleptin therapy, dietary control with additional medication, and psychosocial counseling in line with the patients’ stages of growth and development were important in achieving long-term metabolic control of this condition.

https://doi.org/10.1297/cpe.28.139

Disease module: DeepOracle (Open Targets). Structures: RDKit from PubChem SMILES. Literature: retrieved by DeepSearch across 234,678,978 indexed works (targeted per-candidate search), resolved on OpenAlex.

DeCure is a research and publication project, not medical advice and not a treatment. "DeCure for X" describes a research goal, not a claim that a treatment exists. Committing $DECURE to a DeCure is a vote on research priority — it is not an investment, and confers no royalty, equity or IP ownership. Papers are published open-access.